OCTAPHARMA

Octapharma Underlines Strong Commitment to Haemophilia A Patients with the Publication of New Clinical Data on PK-guided Personalized Prophylaxis According to the NuPreviq Approach with Nuwiq®

Octapharma announces positive new clinical data in 66 previously treated
patients (PTPs) with severe haemophilia A (HA) undergoing PK-guided
personalized prophylaxis with Nuwiq®
according to the
NuPreviq Approach. Data from the NuPreviq study (GENA-21,
NCT01863758)
were published online in April 2017 as an Early View
article in
the internationally renowned medical journal, Haemophilia
.

The NuPreviq study was initiated in August 2013 with the aim of
developing a fully personalized service for HA patients undergoing
prophylactic therapy. Octapharma’s aim is to improve patient treatment
experiences by recognizing that no two HA patients are the same,
therefore a “one size fits all
” approach to prophylaxis is not
ideal. The NuPreviq study investigated a novel approach, allowing the
Nuwiq®
treatment regimen to be adapted to the specific needs
of each individual patient. The NuPreviq Approach measures and profiles
each individual patient’s pharmacokinetic response to treatment, which
then allows the dosing of Nuwiq®
to be personalized according
to the specific circumstances of that patient.

The results of the study showed that this personalized prophylaxis
approach was very effective at preventing bleeds, while reducing the
frequency of dosing and the amount of Nuwiq®
needed. Over 80%
of patients did not experience a spontaneous bleed during the 6 months
of personalized prophylaxis with Nuwiq®
. More than half of
the patients received infusions twice a week or less, and dose
requirements were reduced by 7%. An ongoing extension study (GENA-21b,
NCT02256917) will further evaluate the long-term efficacy and safety of
personalized prophylaxis with Nuwiq®
.

Larisa Belyanskaya, Head of IBU Haematology at Octapharma, said: “We
are very excited by the results of the NuPreviq study and pleased that
the GENA-21 study data are now available to the wider public in the
specialist peer-reviewed publication, Haemophilia. The study shows that
personalized prophylaxis with Nuwiq® provides
excellent bleeding protection, extension of the dosing interval for most
patients, and a reduction in dose. These data further differentiate Nuwiq®
from other products on the market.”

Olaf Walter, Board Member at Octapharma, commented that “Octapharma’s
principal motivation lies with providing dedicated service to our
patients. Developing a custom care package which offers flexible FVIII
therapy
, truly personalized to suit their needs and lifestyle,
has long been a goal of the company and we celebrate today this landmark
publication of the extremely positive NuPreviq clinical data.”

Octapharma would like to thank everyone involved in the study, in
particular the patients and their families, without whom this research
would not be possible.

About GENA-21 and GENA-21b studies:
The GENA-21 and GENA-21b
studies, initiated in August 2013 and March 2015, respectively, are
prospective, open-label, multicentre, Phase 3b studies assessing the
safety and efficacy of PK-guided personalized prophylaxis with Nuwiq®
in previously treated adult patients with severe HA. The studies are
each conducted across 29 study locations worldwide.

About Nuwiq®:
Nuwiq®
is a
naturally long-acting, 4th
generation rFVIII protein,
produced in a human cell line without chemical modification or fusion
with any other protein. Nuwiq®
is cultured without additives
of human or animal origin, is devoid of antigenic non-human protein
epitopes and has a high affinity for the von Willebrand coagulation
factor. Nuwiq®
treatment has been assessed in seven completed
clinical trials which included 201 PTPs (190 individuals) with severe
HA, including 59 children. Nuwiq®
is approved for use in the
treatment and prophylaxis of bleeding across all age groups of PTPs with
haemophilia A in the EU, US, Canada, Australia, Latin America and
Russia. Further worldwide submissions for Nuwiq®
are planned.

About haemophilia A:
Haemophilia A is an X-linked hereditary
disorder caused by FVIII deficiency which, if left untreated, leads to
haemorrhages in muscles and joints and consequently to arthropathy and
severe morbidity. FVIII replacement prophylactic treatment reduces the
number of bleeding episodes and the risk of permanent joint damage. This
disorder affects one in every 5,000 to 10,000 men worldwide. Globally,
75% of haemophilia cases are left undiagnosed or untreated. The
development of neutralizing FVIII antibodies (FVIII inhibitors) against
infused FVIII represents the most serious treatment complication. The
cumulative risk of FVIII inhibitor development is reported to be
currently up to 39%.

About Octapharma
:

Headquartered in Lachen, Switzerland, Octapharma is one of the largest
human protein manufacturers in the world, developing and producing human
proteins from human plasma and human cell lines. As a family-owned
company, Octapharma believes in investing to make a difference in
people’s lives and has been doing so since 1983; because it’s in our
blood.

In 2016, the Group achieved €1.6 billion in revenue, an operating income
of €383 million and invested €249 million to ensure future prosperity.
Octapharma employs more than 7,100 people worldwide to support the
treatment of patients in 113 countries with products across three
therapeutic areas:

Haematology (coagulation disorders)

Immunotherapy (immune disorders)

Critical care

Octapharma owns six state-of-the-art production facilities in Austria,
France, Germany, Mexico and Sweden.